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Quick Info
Ongoing, But Not Recruiting
Trial Type
Treatment Type
Drug Trials
Not Available
Start Date
Contact Information
    Contact information unknown.
Belgium, Other
VIB-KU Leuven Center for Brain & Disease Research, Leuven, 3000, Belgium
France, Other
CHU Lille - CMRR Hôpital Roger Salengro, Lille, 59037, France
CHU Dupuytren, Limoges, Limoges, 87042, France
Groupe Hospitalier Pitie-Salpetriere - La Federation de Maladies du Systeme Nerveux, Paris, 75013, France
Germany, Other
Universitaetsklinikum Ulm - Klinik fuer Neurologie, Ulm, 89081, Germany
Spain, Other
Hospital de la Santa Creu i Sant Pau, Barcelona, 08025, Spain
Hospital Universitario Vall d'Hebron, Barcelona, 08035ES, Spain
Complejo Hospitalario Universitario de Santiago (CHUS), Santiago de Compostela, 15706ES, Spain
Hospital Universitari I Politècnic La Fe, Valencia, 46026, Spain
United States, California
University of California San Diego, La Jolla, CA, 92037, United States
University of California Irvine - ALS & Neuromuscular Center, Orange, CA, 92868, United States
UCSF Neurosciences Clinical Research Unit (NCRU), San Francisco, CA, 94158, United States
United States, Maryland
John Hopkins University, Baltimore, MD, 21287, United States
Johns Hopkins Outpatient Center, Baltimore, MD, 21287, United States
United States, Massachusetts
Massachusetts General Hospital (MGH) - Amyotrophic Lateral Sclerosis (ALS) Multidisciplinary Clinic, Boston, MA, 02114, United States
United States, Minnesota
Mayo Family Clinic Northwest, Rochester, MN, 55905, United States
United States, New York
Hospital for Special Surgery, New York, NY, 10021, United States
Columbia University Medical Center - The Neurological Institute of New York, New York, NY, 10032, United States
United States, North Carolina
The University of North Carolina at Chapel Hill, Department of Neurology, Chapel Hill, NC, 27599, United States
Enrollment Criteria
Breathing Ability
Percent lung function (FVC) or (SVC)
>60% (SVC)
Months Since Onset
Number of months since first symptoms of ALS.
<3 Years
Non-Invasive Ventilation (NIV)
Can PALS use a BiPAP in the trial?
Diaphragm Pacer (DPS)
Can PALS use a DPS in the trial?
Edaravone Usage
Can a PALS use edaravone (Radicut/Radicava) while enrolled in the trial?
Open Label
Update Notes
Study no longer recruiting
No significant updates
Site removed
Sites added, protocol updates
Sites added
Sites added
Study recruiting
Outcome measures updates

Other Information

This is a Phase 2a study to assess the the safety and tolerability of TPN-101 in patients with Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD) Associated with Hexanucleotide Repeat Expansion in the C9orf72 gene (C9ORF72 ALS/FTD).
Inclusion Criteria:
- Documentation of a clinical genetic test demonstrating a hexanucleotide repeat expansion (HRE) in the C9orf72 gene - Has a reliable caregiver/informant to accompany the patient to all study visits For patients with ALS (with or without FTD):
- Diagnosis of ALS (probable, possible, laboratory-supported probable or definite) according to the World Federation of Neurology revised E1 Escorial criteria - Onset of weakness within 3 years prior to Screening - Slow vital capacity (SVC) ≥ 60% of predicted normal adjusted for sex, age, and height (from the sitting position) - Able to perform reproducible pulmonary function tests.
- ALS Functional Rating Scale-Revised (ALSFRS-R) ≥ 30 and score of 3 or 4 on Item #3 (swallowing) at Screening For patients with FTD:
- A gradual, progressive decline in behavior, language, or motor function consistent with mild cognitive impairment, mild behavioral impairment, mild cognitive/behavioral impairment, behavioral variant FTD, primary progressive aphasia, or amnestic syndrome - CDR Dementia Staging Instrument plus National Alzheimer's Coordinating Center Behavior and Language Domains (CDR plus NACC FTLD) global score of 0.5-2.0 at Screening Exclusion Criteria:
- Presence of other significant neurological or psychiatric disorders - History of clinically significant brain abnormality - Clinically significant medical illness - Tracheostomy or diaphragmatic pacing - Autoimmune disease requiring treatment or management (quiescent rheumatoid arthritis, psoriasis, or controlled Type 1 diabetes are acceptable) - History of human immunodeficiency virus (HIV) or hepatitis B infection, or any active infection during Screening, unless the patient will have been symptom-free for at least 30 days prior to randomization
This is a Phase 2a multi-center, randomized, double-blind, placebo-controlled parallel-group, 2-arm study with a long-term, open-label treatment phase in patients with C9ORF72 ALS and/or FTD. This study includes a 6-week Screening Period, a 24-week Double-blind Treatment Period, a 24-week Open-label Treatment Period, and a Follow-up Visit 4 weeks post-treatment.
  • Transposon Therapeutics, Inc.
Trial Protocol as Published on
NCT04993755 (First Published: 7/19/2021)